BOCA RATON, Fla. — The FDA approved the first two PCSK9 inhibitors for use in patients with familial hypercholesterolemia and atherosclerotic CVD who require additional LDL lowering over a year ago, but cost and coverage remain barriers to access for patients and health care providers. A town hall forum held during the American Society for Preventive Cardiology’s Congress on Atherosclerotic Cardiovascular Disease Prevention brought together experts and patients to raise awareness and discuss barriers and potential solutions for the future.
The FDA approved alirocumab injection (Praluent, Sanofi/Regeneron) in July 2015 for use in addition to diet and maximally tolerated statin therapy in adults with heterozygous familial hypercholesterolemia (FH) or patients with clinical atherosclerotic CVD (ASCVD) who require additional LDL lowering and approved evolocumab injection (Repatha, Amgen) in August 2015 for the same indication as well as treatment of patients with homozygous FH.
As new therapeutic options become available,, treatment protocols must be developed based on indications of the FDA approval as well as national guidelines and standards. However, questions arise regarding what to treat, how to treat and by whom patients should be treated, leading to gaps in translation and variances in policy and access, according to the ASPC. In the case of atherosclerotic CVD (ASCVD), patients with severe ASCVD and FH are being denied by payers for most prescriptions written for PCSK9 inhibitors, which has led to access issues and delayed care for patients who would benefit most from this therapeutic option, according to the ASPC.
“Patients are inappropriately being denied a revolutionary advance in lipid management, potentially costing them their lives,” Seth J. Baum, MD, FASPC, FACC, FACPM, FAHA, FNLA, president of the American Society for Preventive Cardiology (ASPC); medical director of Women’s Preventive Cardiology at the Christine E. Lynn Women’s Health and Wellness Institute at Boca Raton Regional Hospital; affiliate associate professor of clinical biomedical science in the department of integrated medicines at Florida State University’s Charles E. Schmidt College of Medicine; and founder and chief medical officer of Excel Medical Clinical Trials, LLC, told the audience.
Seth J. Baum
According to Baum, “80% to 90% of PCSK9 inhibitor prescriptions have been initially denied” by insurance providers since their approval. He noted that “after extensive appeals, only 26.7% of privately insured patients and 46.4% of Medicare beneficiaries obtain approval for these doctors’ prescribed PCSK9 inhibitors.”
Katherine Wilemon, founder and CEO of the FH Foundation, cited new unpublished data from the FH Optimal Care of the US (FOCUS) Survey, showing that 29% of respondents noted that an insurance provider denied coverage of an FH treatment within the past 12 months; 56% noted that they had not received a denial and 15% were unsure. Of the 29% who reported denied overage, 72% of the therapies denied were PCSK9 inhibitors, she told the audience.
Overall, the denials are “capricious,” Baum said. Step therapy requirements are different for each insurance group; payer applications are inconsistent; some providers require Simon Broome criteria to be met for a ‘definite’,- not even ‘probable’ - diagnosis of FH; and some providers require genetic testing vs. clinical diagnosis, according to Baum. Moreover, using North Carolina as an example, Baum noted that second appeals now cost health care providers $250 under Blue Cross Blue Shield of North Carolina.
“We are living in a time where we have many more millions of people insured under the Affordable Care Act. All of my patients are experiencing profound escalations in their premium costs as well as their deductible costs. My patients are being denied not just PCSK9 inhibitors, but routine lab testing,” Peter Toth, MD, PhD, FASPC, congress co-chair and director of preventive cardiology at CGH Medical Center, Sperling, Illinois, said.
A main barrier for patients is the cost of the injectable therapies.
“The patient thinks so much about cost. It is difficult for patients who understand we have a drug (PCSK9 inhibitor) that can help them out, but they just can’t afford it,” Joyce Ross, MSN, CRNP, FNLA, president of the National Lipid Association (NLA) and a consultative education specialist in cardiovascular risk intervention with the University of Pennsylvania Health System, said.
According to Baum, the Institute for Clinical and Economic Review (ICER) initially predicted that PCSK9 inhibitor therapy would cost $7.2 billion in the first year after approval. However, he said, “costs have barely breached $80 million, [which is] 1.2% of the ICER estimate.”
For Wilemon, the “biggest barrier heretofore for the FH population has been lack of diagnosis. There has been a dearth of information about the FH population.”
FH is an inherited disorder that leads to aggressive and premature CVD. It is estimated that one in 250 people worldwide have FH. In the United States, an estimated 1.3 million people have FH, but only 10% are diagnosed. Moreover, more than 90% of people with FH have not been properly diagnosed, according to estimates from the FH Foundation.
The FH Foundation’s stance on PCSK9 inhibitor access is “therapeutically agnostic,” Wilemon said. “We will take any safe, efficacious medication we can to lower our LDL.”
Attendees of the town hall forum also focused on solutions to improve access of PCSK9 inhibitors. One solution, Baum said, is to “allow doctors to do what they have been trained to do and simply prescribe [based] on the label.” He cited broad PCSK9 inhibitor product information as a reason to back up greater prescription based on the label. He also suggested moving away from certain value calculations that “overemphasize and overestimate cost and underemphasize and undervalue benefit.” According to Baum, the goal should be to “shift the priority back to the patient, not the payer.”
Ross, a nurse practitioner, also stressed the importance of the allied health professional in improving access to PCSK9 inhibitors. “It is critically important that [all of] the people in the office have a good handle on exactly what a PCSK9 inhibitor is and exactly why the provider wanted [the patient] to have it. … As we move forward with [prescribing PCSK9 inhibitors], we need to include every person on our team to get in there and fight [to increase access],” she said.
Speakers during the town hall forum, including representatives from the American Association of Clinical Endocrinologists and the American College of Cardiology, in addition to the ASPC, FH Foundation and NLA, also stressed the availability of association guidelines for further guidance on management of these challenging patient populations, in light of barriers to PCSK9 inhibitor access.
This event marked the first of two town hall meetings on access to PCSK9 inhibitors. The second town hall will be held in November during the American Heart Association Scientific Sessions. Then, the representatives from various associations will write and publish a joint paper, according to Baum.
Data from CV outcomes trials of PCSK9 inhibitors are also awaited, with the hope that it may relieve some of these barriers to access in the future.
Several patients attended the open town hall forum to share their stories with FH and ASCVD, access to PCSK9 inhibitor therapy and challenges with insurance providers.
“Time is very valuable for people at very high risk,” Baum said. “… Patients have the power to make a difference and become their own advocates and work with the physicians.”
Baum SJ, et al. Unraveling a Therapeutic Conundrum: A Town Hall on Barriers to Access PCSK9 Inhibitors. Presented at: American Society for Preventive Cardiology Congress on Atherosclerotic Cardiovascular Disease Prevention; Sept. 16-18, 2016; Boca Raton, Fla.
FH Foundation. thefhfoundation.org.
Disclosure: Baum reports serving on speaker’s bureaus for Amgen, AstraZeneca, Cleveland Heart Lab, diaDexus, Genzyme, Kowa Pharmaceuticals, Merck, New Haven Pharmaceuticals, Regeneron and Sanofi and consulting for the aforementioned companies in addition to GLG Group. Toth reports consulting for Amarin, Amgen, AstraZeneca, Kowa Pharmaceuticals, Merck, Regeneron and Sanofi and serving on speaker’s bureaus for Amarin, Amgen, Kowa Pharmaceuticals, Merck, Regeneron and Sanofi. Wilemon is founder and CEO of the FH Foundation. Cardiology Today could not confirm relevant financial disclosures for Ross.